Pharmacists and rare diseases: The results of a survey

Orphan drugs face important challenges in terms of R&D (Research and Development), assessment, production processes, distribution and prescription. Most of the worldwide policies have been focusing on actions aimed at favouring R&D and accelerating access to the market, through orphan designation. Minor attention has been posed on professionals, rare diseases and orphan drugs. This article discusses the role of pharmacists in managing rare diseases and drugs for rare diseases (that are not necessarily orphan). This topic has been scrutinised through a structured questionnaire self-administered to the relevant interviewed: responders represent 27% of total health care organisations. Evidence shows that (i) pharmacists perceive to have an important role in managing rare diseases, (ii) one of the major problems facing pharmacists is the absence or the poorness of clinical pathways for rare diseases, whereas (iii) drug costs and the way drugs are distributed are less important. However, whereas in the Northern part of Italy pharmacists perceive that better organisation and integration among services could improve the situation, in the Central and, especially, Southern Regions pharmacists tend to rely on (and ask for) more regulation; hence, the latter implicitly reveal a more bureaucratic approach, than the former.